Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy

Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy

Status

UNKNOWN

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01610440

Enrollment

Registered

2012-06-04

Start date

2011-10-31

Completion date

2013-10-31

Last updated

2012-11-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophy, Human Umbilical Cord Mesenchymal Stem Cells

Brief summary

Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose motor function gradually and die for heart failure or severe infection at the end stage of DMD. At present, the treatment strategy relies on heteropathy accompanied with rehabilitation training. However, the therapeutic effect remains extremely limited. Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor function, increase muscle strength and reduce abnormal levels of related enzymes, such as creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of hUC-MSCs transplantation for DMD.

Detailed description

This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.

Interventions

BIOLOGICALhuman umbilical cord mesenchymal stem cells

rehabilitation therapy plus human umbilical cord mesenchymal stem cells

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

5 Years to 12 Years

Healthy volunteers

Inclusion criteria

* Aged 5-12 years * Clinical manifestation, enzymology, electromyogram, gene type confirmed the diagnose of Duchenne muscular dystrophy * Sign the consent form and follow the clinic trail procedure

Exclusion criteria

* Not Duchenne muscular dystrophy * Any history of hypersensitivity to serum products,or other know drug and food allergy * Combined Pneumonia or other Severe systemic bacteria infection * HIV+, TPPA +， patients diagnosed as HBV or HCV * Tumor Markers + * Severe psychotic patients, cognitive dysfunction * Coagulation disorders * Uncontrolled hypertension after treatment，blood pressure≥180mmHg/110 mmHg * Other severe systemic or organic disease * Enrollment in other trials in the last 3 months * Received any stem cell therapy in past 6 months * Other criteria that investigator consider improper for inclusion

Design outcomes

Primary

Measure	Time frame
Activities of Daily Living(ADL)scale	1 year after treatment

Secondary

Measure	Time frame
Change from baseline in CK	1 year after treatment
Change from baseline in LDH	1 year after treatment
Change from baseline in ALT	1 year after treatment
Incidences of Adverse Event and Serious Adverse Event	1 year after treatment
Change from baseline to manual muscle test(MMT)	1 year after treatment
Change from baseline in electromyography(EMG)	1 year after treatment
Change from baseline in AST	1 year after treatment

Countries

China

Contacts

Primary ContactLiqing Yao

yaoliqing98731@yahoo.com.cn

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026